The investigational drug AMO-02 (tideglusib), under development by AMO Pharma, has received U.S. Food and Drug Administration (FDA) fast track designation for the treatment of congenital type 1 myotonic dystrophy (DM1). This designation can speed the review of efficacy and safety data for AMO-02 in people with DM, potentially leading to more rapid regulatory approval.

Drugs that receive fast track designation are eligible for more frequent meetings and written communications with the FDA, accelerated review and priority approval, and rolling New Drug Application review.

In a May 30, 2017 press release, AMO Pharma notes that in cellular and animal models of congenital DM1 and Duchenne muscular dystrophy, as well as in muscle biopsies from patients, activity of a protein called glycogen synthase kinase 3 beta (GSK3ß) has been shown to be increased. Inhibitors of GSK3ß have been shown to correct the activity of RNA binding proteins, such as CUGBP1, in animal models of DM1. AMO-02 is designed to work by inhibiting GSK3ß and has demonstrated preclinical efficacy in transgenic models and tissue samples derived from patients with congenital DM1.

A phase 2 clinical trial currently is underway in the United Kingdom to evaluate the safety and efficacy of AMO-02 in people with congenital and juvenile-onset DM who are between the ages of 16 and 45 years old. For more information, visit ClinicalTrials.gov and enter NCT02858908 into the search box.